Gene Therapy Using Lentiviral TYF Vector to Treat Chronic Granulomatous Disease with Long-Term Follow-up

What this Trial Is About

Researchers are evaluating a gene therapy using a high-safety, high-efficiency self-inactivating lentiviral vector TYF to treat Chronic Granulomatous Disease CGD, a rare inherited disorder causing severe infections due to defects in the NADPH oxidase complex. This Phase III trial aims to assess the safety and effectiveness of the TYF-CGD gene transfer protocol in correcting the defective gene responsible for the disease. Participants receive an infusion of their own hematopoietic stem cells that have been modified with the lentiviral TYF vector carrying the functional gene. The gene-modified cells are infused at a dose of 1 to 10 million cells per kilogram of body weight. The study focuses on evaluating the safety of the gene transfer, immune system recovery, and long-term correction of immune dysfunctions after treatment. During the study, participants will be closely monitored for overall survival and gene marking in bone marrow cells over a 15-year follow-up period. Researchers will also track infection frequency and immune function recovery at one year post-treatment through clinical history, physical exams, and laboratory tests. The total duration of participation includes long-term safety and efficacy monitoring extending up to 15 years after receiving the gene therapy.

Lentiviral Gene Therapy for CGD